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Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05906953
Recruitment Status : Recruiting
First Posted : June 18, 2023
Last Update Posted : November 13, 2023
Sponsor:
Collaborator:
Cholgene Therapeutics, Inc.
Information provided by (Responsible Party):
HuidaGene Therapeutics Co., Ltd.

Study Description
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Brief Summary:
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.

Condition or disease Intervention/treatment Phase
Leber Congenital Amaurosis Inherited Retinal Diseases Caused by RPE65 Mutations Drug: HG004 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects With RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)
Actual Study Start Date : October 31, 2023
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: HG004 Drug: HG004
Low dose Medium dose High dose


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of ocular and non-ocular adverse events [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  1. Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart [ Time Frame: 52 weeks ]
  2. Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2. [ Time Frame: 52 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   6 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or females between 6 and 50 years of age at the time of signing the informed consent form.
  • Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.
  • Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations.
  • Ability to perform tests of visual and retinal function.
  • Visual acuity of ≤ 20/80 or visual field less than 20 degrees in the eye to be injected.
  • Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria:

  • Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g., glaucoma requiring upcoming surgery, corneal or significant lenticular opacities).
  • Presence of epiretinal membrane by OCT.
  • Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
  • Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.
  • Prior ocular surgery within six months.
  • Prior gene therapy or oligonucleotide therapy treatments.
  • Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05906953


Contacts
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Contact: Study Director +862125076143 HG00402@huidagene.com

Locations
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United States, California
Research Site Not yet recruiting
Sacramento, California, United States, 95817
Contact: Study Director       HG00402@huidagene.com   
United States, Texas
Research Site Not yet recruiting
Houston, Texas, United States, 77707
Contact: Study Director       HG00402@huidagene.com   
China, Shanghai
Research Site Recruiting
Shanghai, Shanghai, China
Contact: Study Director       HG00402@huidagene.com   
Sponsors and Collaborators
HuidaGene Therapeutics Co., Ltd.
Cholgene Therapeutics, Inc.
Investigators
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Study Director: Study Director HuidaGene Therapeutics Co., Ltd.
More Information
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Responsible Party: HuidaGene Therapeutics Co., Ltd.
ClinicalTrials.gov Identifier: NCT05906953    
Other Study ID Numbers: HG00402
First Posted: June 18, 2023    Key Record Dates
Last Update Posted: November 13, 2023
Last Verified: November 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by HuidaGene Therapeutics Co., Ltd.:
IRD
LCA
LCA2
Additional relevant MeSH terms:
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Blindness
Retinal Diseases
Leber Congenital Amaurosis
Eye Diseases
Vision Disorders
 Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Eye Diseases, Hereditary